Clinical trials: Cytopenias
Thrombotic Thrombocytopenic Purpura (TTP)
A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
Thrombotic thrombocytopenic purpura (or TTP for short) is a condition where blood clots form in small blood vessels throughout the body.
- Trial name
- NCT04683003
- Testing
- TAK-755
- Where
- 27 sites in United States, Austria and 9 more
- Volunteers
- 77 people
- Sponsor
- Takeda
Congenital Thrombotic Thrombocytopenic Purpura
Prospective Psychometric Evaluation Study of a Patient-reported Outcomes (PRO) Instrument for Congenital Thrombotic Thrombocytopenic Purpura (cTTP, Upshaw-Schulman Syndrome [USS], Hereditary Thrombotic Thrombocytopenic Purpura [hTTP]
The purpose of this study is to assess the psychometric properties of a recently developed congenital thrombotic thrombocytopenic purpura (cTTP)-specific patient-reported outcomes (PRO) instrument.
- Trial name
- NCT03519672
- Testing
- No intervention
- Where
- 4 sites in United States, United Kingdom
- Volunteers
- 41 people
- Sponsor
- Baxalta now part of Shire
Neutropenia
A Study of Mavorixafor in Participants With Congenital Neutropenia and Chronic Idiopathic Neutropenia Disorders
This is a 2-part study of mavorixafor in participants diagnosed with chronic neutropenia. The main goal of Part 1 (Phase 1b) is to help researchers learn more about how the investigational medicine, mavorixafor, impacts people…
- Trial name
- NCT04154488
- Testing
- Mavorixafor
- Where
- 7 sites in United States
- Volunteers
- 32 people
- Sponsor
- X4 Pharmaceuticals
Thrombotic Thrombocytopenic Purpura (TTP)
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by a problem in a gene passed down from parents to children, which affects the body's ability to produce…
- Trial name
- NCT07353099
- Testing
- No Intervention
- Where
- 7 sites in Austria, Germany
- Volunteers
- 50 people
- Sponsor
- Takeda
Atypical Hemolytic Uremic Syndrome (aHUS)
Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
The purpose of the study is to assess the efficacy of ravulizumab to control disease activity in children and adolescents with aHUS who have not previously used a complement inhibitor (complement inhibitor treatment-naïve), as…
- Trial name
- NCT03131219
- Testing
- Ravulizumab
- Where
- 18 sites in United States, Belgium and 5 more
- Volunteers
- 34 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
MBL-Deficient, Neutropenia
Study on Mannan Binding Lectin (MBL) Substitution in MBL-Deficient Children With Chemotherapy-Induced Neutropenia
The pharmacokinetics, and clinical and biological effects of MBL replacement therapy in MBL-deficient children during chemotherapy-induced neutropenia were studied.
- Trial name
- NCT00138736
- Testing
- Mannan Binding Lectin (MBL), Mannan Binding Lectin
- Where
- 2 sites in Netherlands
- Volunteers
- 12 people
- Sponsor
- Prothya Biosolutions
Atypical Hemolytic Uremic Syndrome (aHUS)
The Safety and Efficacy of Eculizumab in Japanese Patients With Atypical Hemolytic Uremic Syndrome (aHUS)
Protocol is intended to characterize the overall safety and tolerability of eculizumab in this population.
- Trial name
- NCT01757431
- Testing
- Eculizumab
- Where
- 2 sites in Japan
- Volunteers
- 2 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Purpura, Thrombocytopenic, Idiopathic
A Long-term Safety Study of Eltrombopag in Pediatric Patients With Chronic Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
This was an open-label Phase III extension study to evaluate the long-term safety of eltrombopag in pediatric patients with chronic ITP who previously participated in study TRA115450.
- Trial name
- NCT02201290
- Testing
- Eltrombopag Tablets, Eltrombopag PfOS
- Where
- 3 sites in Russia
- Volunteers
- 9 people
- Sponsor
- Novartis Pharmaceuticals
Hairy Cell Leukemia
US Post-Marketing Safety Study of Moxetumomab Pasudotox-tdfk (LUMOXITI)
This study is being conducted to satisfy a post-marketing requirement (PMR) to provide evidence characterizing 1) the safety of moxetumomab pasudotox-tdfk in patients who are 65 years of age and older and/or 2) the safety of…
- Trial name
- PROXY (NCT04125290)
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- AstraZeneca
Atypical Hemolytic Uremic Syndrome
aHUS Observational Long Term Follow-Up
There is growing but limited information on the long term clinical status of aHUS patients who have previously received or are continuing to receive treatment with eculizumab.
- Trial name
- LTFU (NCT01522170)
- Where
- 56 sites in United States, Australia and 10 more
- Volunteers
- 94 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Ultomiris-exposed Pregnant/ Postpartum, Pregnancy
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of…
- Conditions
- Ultomiris-exposed Pregnant/ Postpartum, Pregnancy, Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS)
- Trial name
- NCT06312644
- Testing
- Ultomiris
- Where
- 7 sites in United States, Australia and 5 more
- Volunteers
- 75 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Atypical Hemolytic Uremic Syndrome
Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment
This was a prospective, open-label study with no participant randomization. Treatment for aHUS was observational and at the discretion of the treating physician.
- Trial name
- EVIDENCE (NCT02614898)
- Testing
- Eculizumab
- Where
- 24 sites in United States, Australia and 2 more
- Volunteers
- 67 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hospitalized Cases of Angioneurotic Oedema, Pneumonia
Drug Utilization and Safety Events Among Children Using Esomeprazole, Other Proton Pump Inhibitors or H2-receptor Antagonists
The purpose of this study is 1. To describe patient characteristics and drug usage among children that are prescribed esomeprazole for the first time and to compare them with patients who are prescribed other proton pump…
- Conditions
- Hospitalized Cases of Angioneurotic Oedema, Pneumonia, Gastroenteritis, Failure to Thrive
- Trial name
- NCT01338363
- Where
- Netherlands
- Volunteers
- 23,470 people
- Sponsor
- AstraZeneca
Neutropenia, Fever
MK0991 Versus Amphotericin B for Empirical Therapy in Febrile, Neutropenic Pediatric Patients (0991-044)
This study is a double-blind, randomized study of MK0991 versus liposomal amphotericin B in the empirical treatment of pediatric patients (ages 2 through 17 years) who have an absolute neutrophil count (ANC) below 500/microliter…
- Trial name
- NCT00082537
- Testing
- caspofungin acetate, Comparator: AmBisome
- Volunteers
- 100 people
- Sponsor
- Merck Sharp & Dohme LLC
Purpura, Thrombocytopenic, Idiopathic
Rapid Infusion Of Immune Globulin Intravenous (IGIV) In Patients With ITP
The objective of this study is to determine if the safety and tolerability of Immune Globulin Intravenous (Human), 10% Caprylate/Chromatograph Purified (IGIV-C) is similar when infused at two different infusion rates.
- Trial name
- NCT00220727
- Testing
- Immune Globulin IV [Human], 10% Caprylate/Chromatography Purified
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Grifols Therapeutics LLC
Adenosine Deaminase Deficiency
Investigation of ADA Enzyme Deficiency
This observational study was designed as a prospective epidemiological screening study. Patients who applied to the centers participating in the study and were found to be lymphopenic in at least one examination will be included…
- Trial name
- NCT05300334
- Testing
- adenosine deaminase test
- Where
- Turkey (Türkiye)
- Volunteers
- 800 people
- Sponsor
- TRPHARM
Sarcoma, Neutropenia
Pegfilgrastim as Support to Pediatric Sarcoma Patients Receiving Chemotherapy
Physicians are conducting a clinical trial for patients with pediatric sarcoma. Sarcoma is a type of bone cancer that can be treated with chemotherapy which can affect the bone marrow, where blood cells are produced.
- Trial name
- NCT00035620
- Testing
- pegfilgrastim, filgrastim
- Volunteers
- 44 people
- Sponsor
- Amgen
Thrombocytopenia
Efficacy and Safety Study of Platelets Treated for Pathogen Inactivation and Stored for Up to Seven Days
Objective: To determine if platelets treated for pathogen inactivation and stored for 6 to 7 days are safe and effective compared to platelets collected by the same method, stored for the same amount of time and not treated for…
- Trial name
- TESSI (NCT00261924)
- Testing
- Transfusion of Pathogen Inactivated Platelets stored for 6-7 days
- Where
- United Kingdom
- Volunteers
- 211 people
- Sponsor
- Cerus Corporation
Neutropenia
Pegfilgrastim as Support to Advanced Breast Cancer Patients Receiving Chemotherapy
Physicians are conducting a clinical trial for patients with advanced breast cancer. Breast cancer can be treated with chemotherapy which can affect the bone marrow, where blood cells are produced.
- Trial name
- NCT00035594
- Testing
- pegfilgrastim
- Volunteers
- 928 people
- Sponsor
- Amgen